The drug lomitapide was originally developed to reduce low-density lipids (“bad” cholesterol) but failed in Phase I clinical trials. M. Beer and colleagues at Aegerion Pharmaceuticals (Cambridge, MA), however, “rescued” lomitapide by developing it as a treatment for the orphan disease homozygous familial hypercholesterolemia (HoFH), a genetic disorder that affects the liver and often causes heart failure. Lomitapide inhibits the microsomal triglyceride transfer protein needed for the assembly of bad cholesterol and its secretion in the liver. Aegerion and its pharmaceutical services partner, Aptuit (Greenwich, CT), rushed lomitapide through trials; and the US FDA approved as an HoFH drug.
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